FDA Panel Clears Drug for Use Before Breast Cancer Surgery

A federal advisory committee cleared the way for a use of a cancer drug to treat patients before surgery to remove their tumors. The FDA’s Oncologic Drugs Advisory Committee voted 13 to 0, with one abstention, that Perjeta, a drug approved last year for late-stage breast cancer, could also be used at the earliest stage of the disease. The belief is that the drug will shrink tumors in the pre-surgical environ, making some otherwise inoperable tumors easier to remove.

Perjeta, also known as pertuzumab, is designed for patients whose tumors contain excess levels of the HER-2 protein and was developed by Roche’s Genentech unit.

Evidence Supporting the Decision

The FDA concluded that the totality of the evidence, including the drug’s relative safety in advanced breast cancer, tilted their decision in favor of approval despite the small trial size. The trial was composed of a 417 woman study comparing Perjeta in different combinations against older breast cancer treatments. When Perjeta was combined with Genentech drug, Herceptin, and standard chemotherapy, 39 percent of women saw their cancer reach undetectable levels. Only 21 percent of women experienced the same results from taking Herceptin and chemotherapy alone. After drug treatment, all the women received standard breast surgery to remove any tumors.

Insight into the Approval Process

In addition to the approval of Perjeta for pre-surgical use, the decision of the advisory panel offers insight into quicker approvals of other cancer drugs. In background memos prior to the advisory committee’s meeting, the implication was that the FDA was ready to support “accelerated approval” because of earlier studies and use of Perjeta. Accelerated approval could take years off the traditional development process for cancer drugs. For instance, Herceptin was approved in 1998 for use in advanced breast cancer and wasn’t approved for use in early-stage cancer until 2006. In contrast, Perjeta was first approved last year for advanced cancer cases and now could be approved for early use by the end of October. Although the approval does accelerate delivery to market, the FDA made clear that it considered the Perjeta case unique in that the drug is already used and has a track record. Most drugs will not be approved based on such limited data.

Post-Market Study

As required by the FDA, Genentech will conduct a follow-up trial using 4,800 patients who have already had surgery. The FDA has expressed some concern that Perjeta, in combination with anthracycline chemotherapies could cause heart problems. The FDA advisory panel expressed similar sentiment and recommended that the FDA to carefully scrutinize the results of the follow-up trial to ensure they support the benefit to patients suggested by the small trial.